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AURORA, Colo.--(BUSINESS WIRE)--Ocugen,
Inc. and the University
of Colorado today announced exclusive license agreements that allow
for Ocugen to continue developing two drug candidates for the treatment
for ophthalmology indications, and that one of the assets, OCU100,
recombinant lens epithelium derived growth factor 1-326 (LEDGF1-326),
received orphan-drug status from the U.S. Food and Drug Administration
for treatment of retinitis pigmentosa (RP), a rare eye disease.

“Orphan drug designation from the FDA’s Office of Orphan Products
Development is a significant milestone that will allow Ocugen to
accelerate the clinical development of OCU100, which has the potential
to be the first approved therapeutic for retinitis pigmentosa,” said
Shankar Musunuri, PhD, MBA, founder and chairman of the Ocugen Board of
Directors.

Ocugen scientific founder and board member Uday Kompella, PhD, a
professor of Pharmaceutical Sciences, Ophthalmology and Bioengineering
at CU’s Anschutz Medical Campus, is the inventor of OCU100. Ocugen
licensed all assets related to LEDGF, including LEDGF1-326 and OCU200,
an anti-angiogenic tumstatin fusion protein, to be developed for
treatment of wet age-related macular degeneration (AMD) from the
University of Colorado in March.

Retinitis pigmentosa is a rare eye disease caused by inherited gene
mutations that lead to retinal degeneration affecting approximately
100,000 people in the United States, according to the Foundation
Fighting Blindness. People with RP experience a gradual decline in their
vision because photoreceptor cells in the retina die. It is a
progressive disorder, and most people with RP are legally blind by age
40. There is no FDA approved therapeutic for RP.

“OCU100 has shown the potential as a promising therapeutic agent for
treating retinitis pigmentosa by reducing protein aggregation and
associated cellular stresses, which are known to contribute to this
condition,” said Dr. Kompella. “With impressive preclinical data, we
look forward to progressing with a phase 1 study for safety and
tolerability in patients sometime in 2015.”

Dr. Kompella said a variety of mutations, including P23H mutation in
rhodopsin, a critical protein in the retina that is responsible for
vision, have been linked to the development of RP. P23H rhodopsin is
known to form large clusters or aggregates within retinal cells, leading
to cellular stress and ultimately cell death. OCU100 with its ability to
rescue retinal cells from protein aggregation and associated stresses,
has the potential to revolutionize the treatment of RP.

About Orphan Drug Designation

FDA Office of Orphan Products Development (OOPD) grants orphan
designation for novel drugs or biologics that treat a rare disease or
condition affecting fewer than 200,000 patients in the U.S. Orphan
designation qualifies the sponsor of the drug for various development
incentives of the Orphan Drug Act (ODA) including seven-year period of
U.S. marketing exclusivity, tax credits for qualified clinical testing,
waiver of prescription drug user fee for marketing application, and
ability to apply for grants. The OOPD also works on rare disease issues
with the medical and research communities, professional organizations,
academia, governmental agencies, industry, and rare disease patient
groups.

About Ocugen, Inc.

Ocugen is advancing novel biologicals discovered based on endogenous
proteins with well understood biology at the molecular, cellular, and
whole animal level to treat eye diseases. The therapeutic proteins in
the pipeline are derived from cell survival factors such as lens
epithelium derived growth factor (LEDGF) and anti-angiogenic proteins
such as tumstatin. The intellectual property covers a variety of related
protein constructs including fusion proteins with superior activity.

About the University of Colorado

The University of Colorado is a premier public research university with
four campuses: the University of Colorado Boulder, the University of
Colorado Colorado Springs, the University of Colorado Denver and the
University of Colorado Anschutz Medical Campus. Some 57,591 students are
pursuing academic degrees at CU. Academic prestige is marked by the
university’s five Nobel laureates, eight MacArthur “genius” Fellows, 18
alumni astronauts and 19 Rhodes Scholars. For more information about the
entire CU system, and to access campus resources, go to www.cu.edu.